Patent classifications
A61K38/1719
Methods for Generation of Cell-derived Microfilament Network
This invention provides for a network of cell-derived microfilaments. Also provided are methods of producing a network of microfilaments via culturing cells in a matrix support and cell culture medium wherein the cells proliferate and form aggregated cell masses, which produce microfilaments external to and surrounding the cell masses, and wherein the extracellular microfilaments connect and form a continuous extracellular microfilament network, and methods for treating a medical condition as well as facilitating wound repair and tissue regeneration comprising applying the microfilament network to an area in need of treatment.
Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
Compositions, Methods and Uses of a Teneurin C-Terminal Associated Peptide-1 (TCAP-1) for Treating Opioid Addiction
The present matter relates to compositions comprising a Teneurin C-terminal Associated Peptide-1 (TCAP-1), or a pharmaceutically acceptable salt or ester thereof or a pharmaceutical composition comprising same and methods and uses of same for preventing and/or treating opioid addiction.
Antimicrobial biopolymer compositions, methods of synthesis, and applications of use
Biopolymer compositions comprising antimicrobial peptides (AMPs) for treating infections such as bacterial infections, viral infections, fungal infections, and parasitic infections. The compositions herein may also be used for treating infections associated with antibiotic-resistant bacteria, antifungal-resistant fungi, antiviral-resistant viruses, or for treating biological warfare agents (BWAs) such as Bacillus anthracis and Yersenia pestis. The present invention also provides methods of synthesis of said biopolymer compositions, wherein AMP biopolymers can be synthesized as an artificially engineered protein by genetically fusing an AMP; a protein that behaves similarly to polymer tethers; and a protein as a modifiable material platform that can transform to self-assembled nanoparticles, self-standing films, or adhesives to easily attach tethered AMPs onto any biomaterial surface for various clinical applications.
COMPOSITIONS AND METHODS FOR USING ACTIN-BASED PEPTIDES TO MODULATE CELLULAR BIOACTIVITY AND CELLULAR SUSCEPTIBILITY TO INTRACELLULAR PATHOGENS
Compositions and methods for using actin-based peptides to modulate cellular bioactivity, including modulation of cellular susceptibility to intracellular pathogens, such as bacteria and viruses.
ARTIFICIAL EXOSOME COMPOSITION AND RELATED METHODS
Novel artificial exosomes and methods for producing novel artificial exosomes are provided. Methods of delivering cargo molecules to a cell using artificial exosomes are also provided.
Methods and compositions for promoting thermogenic potential
The invention provides methods and compositions relating to molecular targets identified as being capable of increasing or decreasing thermogenic potential in cells, including preadipocytes. Included in the invention are methods and compositions relating to inhibiting or suppressing the activity of an uncoupling protein 1 (UCP1) negative regulator, such as cardiac actin 1 (ACTC1), somatostatin receptor 1 (SSTR1), FAT atypical cadherin 1 (FAT1), and protein tyrosine phosphatase receptor type B (PTPRB). Also included in the invention are methods and compositions relating to activating a UCP1 positive regulator, such as phosphatidylinositol-3,4,5-triphosphate-dependent Rac exchange factor 1 (PREX1), cortactin binding protein 2 (CTTNBP2), doublesex and mab-3-related transcription factor-like family A1 (DMRTA1), and endothelin receptor type B (ENDRB). The invention also provides methods and compositions relating to enrichment of cells having thermogenic potential based on cell surface markers, e.g., CD29, identified as being predictive of such.
Adeno-associated virus variant capsids and methods of use thereof
Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.
Adeno-associated virus variant capsids and methods of use thereof
Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.
METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53
The invention relates to a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, or exons 50-53 of the DMD pre-mRNA in a patient, the method comprising providing the patient with the molecule. The invention also relates to the molecule as such.